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In this meeting, chaired by Carsten B\u00f6nnemann (NINDS, NIH) and Barry Byrne (University of Florida), academic and industry experts and stakeholders convened to openly discuss adverse events linked to clinical trials, as well as other challenges emerging in preclinical studies associated with difficulties in the translation of AAV-mediated gene therapies.<\/jats:p>","DOI":"10.3233\/jnd-221639","type":"journal-article","created":{"date-parts":[[2023,2,14]],"date-time":"2023-02-14T10:51:16Z","timestamp":1676371876000},"page":"327-336","source":"Crossref","is-referenced-by-count":62,"title":["Meeting Report: 2022 Muscular Dystrophy Association Summit on \u2018Safety and Challenges in Gene Transfer Therapy\u2019"],"prefix":"10.1177","volume":"10","author":[{"given":"Angela","family":"Lek","sequence":"first","affiliation":[{"name":"Muscular Dystrophy Association, Chicago, IL, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Evrim","family":"Atas","sequence":"additional","affiliation":[{"name":"Muscular Dystrophy Association, Chicago, IL, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sharon E.","family":"Hesterlee","sequence":"additional","affiliation":[{"name":"Muscular Dystrophy Association, Chicago, IL, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Barry J.","family":"Byrne","sequence":"additional","affiliation":[{"name":"Powell Gene Therapy Center, University of Florida, Gainesville, FL, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Carsten G.","family":"B\u00f6nnemann","sequence":"additional","affiliation":[{"name":"Neuromuscular and Neurogenetic Disorders of Childhood Section, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, Maryland, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","reference":[{"issue":"6","key":"10.3233\/JND-221639_ref1","doi-asserted-by":"crossref","first-page":"387","DOI":"10.1358\/dot.2021.57.6.3264117","article-title":"Onasemnogene abeparvovec-xioi: A gene replacement strategy for the treatment of infants diagnosed with spinal muscular atrophy","volume":"57","author":"Schwartz","year":"2021","journal-title":"Drugs of Today"},{"issue":"3","key":"10.3233\/JND-221639_ref3","doi-asserted-by":"crossref","first-page":"350","DOI":"10.1016\/j.gim.2022.01.568","article-title":"OP ASPIRO gene therapy trial in X-Linked Myotubular Myopathy (XLMTM): Update on preliminary efficacy and safety findings","volume":"24","author":"Shieh","year":"2022","journal-title":"Genetics in Medicine.S"},{"issue":"1","key":"10.3233\/JND-221639_ref4","doi-asserted-by":"crossref","first-page":"15","DOI":"10.3233\/JND-190426","article-title":"Advancements in AAV-mediated Gene Therapy for Pompe Disease","volume":"7","author":"Salabarria","year":"2020","journal-title":"J Neuromuscul Dis"},{"key":"10.3233\/JND-221639_ref5","unstructured":"Manso AM , Hashem SI , Nelson BC , Gault E , Soto-Hermida A , Villarruel E , et al. 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